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7 results

bivigam
(Immune Globulin Intravenous (Human) 10%)
ADMA Biologics, Inc.
Usage: BIVIGAM is indicated for treating primary humoral immunodeficiency (PI) in adults and children aged 2 years and older. It addresses conditions such as common variable immunodeficiency, X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.
carbaglu
(Carglumic Acid)
Recordati Rare Diseases
Usage: CARBAGLU is indicated for adult and pediatric patients as adjunctive therapy and maintenance treatment for acute and chronic hyperammonemia due to N-acetylglutamate Synthase (NAGS) deficiency, as well as adjunctive therapy for acute hyperammonemia caused by Propionic Acidemia or Methylmalonic Acidemia.
ilaris
(canakinumab)
Novartis Pharmaceuticals Corporation
Usage: ILARIS (canakinumab) is indicated for treating autoinflammatory Periodic Fever Syndromes, including CAPS, TRAPS, HIDS/MKD, and FMF; active Still's Disease in patients 2 years and older; and symptomatic treatment of gout flares in adults when other treatments are unsuitable.
jynarque
(tolvaptan)
Otsuka America Pharmaceutical, Inc.
Usage: JYNARQUE is indicated to slow the decline of kidney function in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD).
kanuma
(Sebelipase alfa)
Alexion Pharmaceuticals, Inc.
Usage: KANUMA® is indicated for treating patients diagnosed with Lysosomal Acid Lipase (LAL) deficiency.
pombiliti
(cipaglucosidase alfa-atga)
AMICUS THERAPEUTICS US, LLC
Usage: POMBILITI, in combination with Opfolda, is indicated for treating adult patients with late-onset Pompe disease (GAA deficiency) weighing ≥40 kg, who are not experiencing improvement on their current enzyme replacement therapy (ERT).
tegsedi
(inotersen)
Akcea Therapeutics, Inc.
Usage: TEGSEDI is indicated for treating the polyneuropathy associated with hereditary transthyretin-mediated amyloidosis in adults.

Genetics & Rare Disease drugs

bivigam

carbaglu

ilaris

jynarque

kanuma

pombiliti

tegsedi